RICHMOND, Calif., Feb. 6, 2018 /PRNewswire/ — Sangamo Therapeutics, Inc. (Nasdaq: SGMO) today announced the presentation of initial safety data from the CHAMPIONS Study, the Phase 1/2 clinical trial evaluating SB-913 for the treatment of mucopolysaccharidosis (MPS) type II, a rare disease also known as Hunter syndrome. The data are being presented this afternoon at the 2018 WORLDSymposium medical congress being held in San Diego.
The poster presentation titled “Update on phase 1/2 clinical trials for MPS I and MPS II using ZFN-mediated in vivo genome editing” includes safety data collected and prepared as of December 27, 2017 and describes the first six weeks of experience of the first patient in the CHAMPIONS study, who was treated November 13, 2017 with a dose of 5.00E+12 vg/kg of SB-913. The patient tolerated the infusion well. Mild (Grade 1) adverse events related to the study drug were reported on the fourth day after dosing as dizziness, weakness, and frequent urination, all of which resolved within one day without treatment. No other adverse events related to the study drug were observed. Liver function tests have remained within normal limits for the patient since the infusion.
One serious adverse event has been reported and is classified as unrelated to SB-913. The subject developed an upper respiratory tract infection and was hospitalized for acute bronchitis. The subject, who recovered after receiving medical treatment, has chronic pulmonary disease and has previously been hospitalized for a similar respiratory illness.
A second patient was treated in the study in mid-January 2018, also at a dose of 5.00E+12 vg/kg.
“Two patients have thus far been treated in the CHAMPIONS Study, and both have tolerated the SB-913 infusions well, with no concerning safety issues related to study drug seen to date,” said Paul Harmatz, M.D., a pediatric gastroenterologist and a principal investigator for the CHAMPIONS study at the UCSF Benioff Children’s Hospital Oakland, where the first subjects in the study were treated.
Dr. Edward Conner, Chief Medical Officer of Sangamo, also commented: “We are pleased with how the CHAMPIONS Study is progressing. We expect enrollment of remaining patients to proceed on schedule and to begin to report additional safety and initial efficacy data by mid-year.”
Sangamo and collaborators are presenting four abstracts at the 2018 WORLDSymposium:
Tuesday, February 6, 4:30 – 6:30pm, Poster Presentations
#134 “Update on phase 1/2 clinical trials for MPS I and MPS II using